Santa Cecilia

Researchers are evaluating the safety and effectiveness of GTX-102, an antisense oligonucleotide, in people with Angelman syndrome, a genetic condition. This Phase 2 open-label basket study includes participants of various genotypes and ages, divided into four subprotocols (A, B, C, and D), to study different forms and age groups affected by Angelman syndrome. The study aims to observe how GTX-102 works and whether it is safe for both children and adults with this condition. The study includes screening, loading, and maintenance periods for all subprotocols. Subprotocols A, B, and C have a single treatment arm where participants receive GTX-102. Subprotocol D randomly assigns participants in a 2:1 ratio to either receive GTX-102 or no treatment, with both groups following the same visit schedule. After completing the study, participants may choose to continue receiving GTX-102 in a long-term extension study. Participants will undergo various assessments including cognitive testing, safety monitoring for adverse events, and response evaluations using specific outcome measures up to day 506. Regular laboratory tests, magnetic resonance imaging (MRI), lumbar punctures, and adherence to scheduled visits and treatments are required. Researchers will track treatment-emergent adverse events and serious adverse events, as well as cognitive and multidomain responses, to better understand the impact of GTX-102 over time.

Researchers are investigating whether the medicine vicadrostat, when taken together with empagliflozin, can lower the risk of heart-related problems in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. This study is a Phase III trial that compares the effects of vicadrostat plus empagliflozin to a placebo plus empagliflozin in people with these conditions. Participants are randomly assigned to one of two groups: one group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets that look like vicadrostat along with empagliflozin. All participants take one tablet daily for a period ranging from two and a half years up to four years and three months. Throughout the study, participants continue their usual medications for diabetes, high blood pressure, and cardiovascular disease. During up to 51 months of participation, participants visit the study site regularly where doctors collect health information and blood samples. Researchers track when participants experience cardiovascular events such as heart-related deaths or heart failure events. The study also monitors participants’ overall health and any side effects they may experience to assess the safety and effects of the treatments.